SAPHO Syndrome
Conditions
Keywords
Treatment, Bisphosphonates, Response rate
Brief summary
This study is designed to evaluate long term efficacy of intravenous bisphosphonates for bone marrow edema in patients with SAPHO syndrome.
Detailed description
The study will evaluate the long term efficacy at 3 days, 3 and 6 months after pamidronate disodium administered. Patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 months (up to month 6).
Interventions
the patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 month (up to month 6)
Sponsors
Peking Union Medical College Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female patients between the ages of 18 and 70 years 2. Only 1 of the 4 inclusion criteria is sufficient to arrive at a diagnosis of SAPHO syndrome： * Osteo-articular manifestations of acne conglobata, acne fulminans, or hidradenitis suppurativa * Osteo-articular manifestations of PPP * Hyperostosis (of the anterior chest wall, limbs or spine) with or without dermatosis * CRMO involving the axial or peripheral skeleton with or without dermatosis Palmoplantar pustulosis(PPP); chronic recurrent multifocal osteomyelitis(CRMO) 3. MRI shows bone marrow edema in affected site in patients 4. Blood serum of patients show the normal white blood cell count, liver and renal function 5. Patients who like to be followed up for 1 years 6. Other protocol defined inclusion criteria may apply
Exclusion criteria
1. Women in pregnancy or lactation. 2. Septic osteomyelitis 3. Infectious chest wall arthritis 4. Infections PPP 5. Palmo-plantar keratodermia 6. DISH except for fortuitous association 7. Osteoarticular manifestations of retinoid therapy 8. Other protocol defined
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| response to the treatment by the change of MRI result at screening, baseline, month 3 and month 6 | 3 and 6 month (up to 6 month) |
Secondary
| Measure | Time frame |
|---|---|
| response to the treatment by the change of bath ankylosing spondylitis functional index (BASFI) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| physician's global assessment of disease activity at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of Visual Analogue ale/Score (VAS) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of bath ankylosing spondylitis metroloty index (BASMI) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of bath ankylosing spondylitis disease activity index (BASDAI) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of erythrocyte sedimentation rate (ESR) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of high sensitivity-C reaction protein (hs-CRP) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of Osteocalcin at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of beta-Crosslaps (beta-CTx) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
| response to the treatment by the change of ankylosing spondylitis disease activity score (ASDAS) at screening, baseline, month 3 and month 6 | 3 days, 3 and 6 month (up to 6 month) |
Outcome results
None listed